OSTEOPETROSIS - THE PHARMACO-PHYSIOLOGICAL BASIS OF THERAPY

Medical treatments of osteopetrosis have attempted to improve hematolo gic function, reduce the osteosclerotic condition, and/or improve immu ne function. Prednisone therapy has improved hematologic function in s ome patients, but has not resulted in a reduction in bone mass. Calciu m deficient diets have limited further sclerosis in some patients. Hig h-dose calcitriol and parathormone infusions have stimulated osteoclas tic activity. In some patients, high-dose calcitriol has resulted in c linical improvement. Newer treatments, such as interferon gamma and ma crophage colony stimulating factor, may alter the osteoclastic and imm une defects by stimulating cellular formation and function. These ther apies, alone or in combination, ameliorate but do not cure the osteope trotic condition.